Report on 'Cell, Gene and Tissue Regulatory Framework in Australia: Stakeholder Perspectives' - TGA response

Terms and Definitions

TGA should align definitions and classifications with international regulators.

Wherever possible, TGA will continue to align definitions and classifications with international regulators, but due to differences in the terminologies across overseas regulators complete harmonization is not possible.

TGA to communicate the rationale behind the perceived intersection between orphan drug status and Class 4 biologicals.

There is currently no orphan drug scheme for any biologicals, irrespective of the Class. Introduction of an orphan drug scheme for biologicals would require legislative changes and financial support for evaluations. This would require a policy decision by Government.

Clinical Trial Pathways

• TGA should enhance communication about when to use Clinical trial notification (CTN) vs Clinical trial approval (CTA) pathways.
• Timelines for CTA process to be published on the TGA website.
• Education around Advanced Therapies for the TGA personnel to support CTA process improvements.
• TGA to continue to work with CORs to ensure other jurisdictions can recognise CTA.

TGA will instigate a review of the CTA process, including the need for an education campaign to raise awareness and clarity of the timeframes and process.

Consultation meetings are available for applicants with new CTAs.

TGA to consider supporting centres in developing capacity to run trials for Advance Therapies.

TGA will explore options on how best to engage with developers of Advanced (cell and tissue) Therapies during early stages of product development. However, capacity building for clinical trial centres is out of scope for TGA's role.

Good Manufacturing Practice (GMP)

• TGA to provide more guidance around when GMP is applicable across the different stages.
• TGA to improve communication between the different branches responsible for the GMP.

TGA to increase guidance on GMP requirements at various stages of cell and tissue therapy development

TGA will continue to investigate options to align GMP requirements with those in the EU PIC/S GMP Guidance.

The TGA digital transformation project will support applicants (and TGA staff) in the collaborative evaluation of products and in advising applicants of the stage of different evaluation processes.

TGA-OGTR interactions

• Opportunity to improve and streamline the interactions between the TGA and OGTR. Many industry and patient advocacy group stakeholders recommending, the TGA should directly interact the OGTR
• TGA to provide more guidance about when industry should interact with the OGTR across each stage of the development cycle.
• TGA to communicate why gene-modified cell therapies are distinct from gene therapies.
• TGA and the OGTR to apply consistent terminology.

TGA and OGTR have instigated a process to review their interactions around GMOs, including a revision of guidance material published on our websites. The two regulators will better communicate the availability of joint pre-submission meetings for applicants.

However, differences in terminology between regulators, such as "dealings" with GMOs are defined in the relevant legislation, are long-established and would be difficult to change.

TGA Registration Pathways

Expedited pathways should be available for all therapies, regardless of their classification and should be based on patient need.

TGA has recently completed a consultation on the potential introduction of a Priority Pathway for biologicals, and a proposal is before government.

TGA to make proactive adaptions to regulatory frameworks and communicate how they use horizon scanning to implement these changes.

This review forms part of an ongoing commitment to constantly review changing industry needs and emerging technologies and consider the need to make proactive amendments, where necessary.

For example, in November 2021 TGA published the outcomes of a review into its use of Real-World Evidence and Patient Reported Outcomes and proposed several actions such as adoption and communication of relevant regulatory guidance. Utilisation of Real-World Evidence will also be communicated to developers of cell and tissue therapies.

TGA to continue and expand International collaboration on regulatory frameworks.

The TGA works closely with other regulators both in comparable and developing health systems. We will continue to support these efforts and look for new opportunities to collaborate with our partners to ensure timely access to new products for Australians.

TGA to gather representative expert advisory views when appraising Advanced Therapies and provide more transparency around discussions with Advisory Committee.

The TGA seeks the advice of the Advisory Committee on Biologicals in the regulatory review of novel cell and tissue therapies and as required engages specialist advisers to broaden available advice on specific products.

Publication of Public Assessment Reports will commence for cell and tissue therapies.

TGA to consider a holistic approach and a single point of entry for data input when working with other agencies in the patient access pathway.

We are continuing to progress our Digital Transformation Program, to streamline our business systems and modernise IT infrastructure. This includes support for the National One Stop Shop and the National Clinical Trials Front Door.

Pharmaceutical Benefits Advisory Committee (PBAC)/Medical Services Advisory Committee (MSAC) interface with TGA

TGA to work more closely with the relevant HTA bodies.

TGA has been working with PBAC and MSAC to increase collaboration and enable parallel processing.

However, only some cell and tissue therapies are considered by either committee, and where it does take place the consideration by MSAC may not be product specific.